Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

Posted on: 20 September 2016, source: India Times
MIT scientists have developed a new gene therapy technique that can stop the spread of malignant cells around the body - the leading cause of mortality in women with breast cancer. The treatment uses microRNAs - small noncoding RNA molecules that regulate gene expression - to control the spread of cancer cells around the body, known as metastasis. The therapy could be used alongside chemotherapy to treat early-stage breast cancer tumours before they spread, according to Natalie Artzi, a principal research scientist at Massachusetts Institute of Technology (MIT) in the US.

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Posted on: 8 September 2016, source: Reuters
Healthcare company Novartis has either fired or reassigned roughly 400 researchers previously tasked with developing cell and gene therapies. While most of those scientists will be employed by the company elsewhere, 120 positions will be eliminated, according to Reuters. The Cell and Gene Therapies Unit, led by Usman Azam, attracted attention for a promising experimental therapy based on chimeric antigen receptor T-cells (CAR-T), called CTL109, designed to treat lymphomas in children, reports BusinessInsider.

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Posted on: 2 September 2016, source: GEN
Door Has Been Opened to a Range of Novel Biological Therapies for Human Diseases. Twenty-seven years after Steven A. Rosenberg, M.D., Ph.D., at the National Cancer Institute Bethesda campus performed the first human gene transfer experiment, gene therapy is poised to take its place in the armamentarium of biologic therapies available to treat human diseases.
Some examples: the EMA-approval in November 2012 of Glybera® (Uniqure), an intramuscular recombinant adeno-associated virus (rAAV) product for treatment of lipoprotein lipase deficiency; and the recently announced Phase II trial results showing efficacy of another rAAV product for a genetic cause of blindness (LCA2)—for which a Biologics Licensing Application is anticipated in the U.S. this year.

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Posted on: 25 August 2016, source: PRNewswire
Data from Vybion on a novel treatment for Huntington's disease has been published in the Journal of Neurodegenerative diseases. The published study links the ability of Vybion's proprietary, novel Intrabody (INT41) blocking of cellular gene dysregulation to the delay of cognitive and motor function loss in the well-validated vR6/2 animal model. INT41 interferes with direct binding of toxic N-terminal huntingtin fragments to DNA, as well as their transport into the nucleus. The data support a direct gain of function of N-terminal huntingtin protein fragments that may lead to neuron dysfunction and brain atrophy as well as a novel therapeutic modality.

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Posted on: 24 July 2016, source: DDD-Mag
AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough Therapy Designation is based on preliminary clinical results from the ongoing trial of AVXS-101, conducted in collaboration with The Research Institute at Nationwide Children's Hospital and The Ohio State University.

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Posted on: 16 July 2016, source: GEN
Zopharm Oncology has disclosed the deaths of three patients in a Phase I study of its gene therapy candidate Ad-RTS-hIL-12 plus oral veledimex for a form of brain cancer. The third patient died of an intracranial hemorrhage that occurred sometime following discharge from a treating center for the Phase I study, Ziopharm said today in a statement. The study is designed to assess the combination of Ad-RTS-hIL-12 with orally administered veledimex in patients with recurrent or progressive glioblastoma (GBM) or grade III malignant glioma.

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Posted on: 1 July 2016, source: Nature
The perils of the past must not be allowed to happen again. Jesse Gelsinger was 18 and healthy when he died in 1999 during a gene-therapy experiment. He had a condition called ornithine transcarbamylase deficiency (OTC), but it was under control through a combination of diet and medication. Like others with the disorder, Gelsinger lacked a functional enzyme involved in breaking down ammonia, a waste product of protein metabolism that becomes toxic when its levels become too high. The gene therapy that he received used a viral vector to introduce a normal gene for the enzyme. Gene therapy remains an obvious route to treat OTC. Simply adding the missing gene has been shown to repair metabolism in mice. But the memory of what happened to Gelsinger has slowed progress in gene therapy for any condition.

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